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Zoecklein, Laurie J

Mayo Clinic

Department of Neurology, Mayo Clinic, Rochester, MN 55905, USA.

Citations 2

Naturally Occurring Monoclonal Antibodies and Their Therapeutic Potential for Neurologic Diseases

JAMA Neurology | September 21, 2015 | Volume: 72 | Issue: 11 | Pages: 1346

10.1001/jamaneurol.2015.2188 | PMID 26389734

A natural human IgM that binds to gangliosides is therapeutic in murine models of amyotrophic lateral sclerosis

Disease Models & Mechanisms | May 29, 2015 | Volume: No volume available | Issue: No issue available | Pages: No page available

10.1242/dmm.020727 | PMID 26035393

Amyotrophic lateral sclerosis (ALS) is a devastating, fatal neurologic disease that primarily affects spinal cord anterior horn cells and their axons for which there is no treatment. Here we report the use of a recombinant natural human IgM that binds to the surface of neurons and supports neurite extension, rHIgM12, as a therapeutic strategy in murine models of human ALS. A single 200 µg intraperitoneal dose of rHIgM12 increases survival in two independent genetic-based mutant SOD1 mouse strains (SOD1G86R and SOD1G93A) by 8 and 10 days, delays the onset of neurologic deficits by 16 days, delays the onset of weight loss by 5 days, and preserves spinal cord axons and anterior horn neurons. Immuno-overlay of thin layer chromatography and surface plasmon resonance show that rHIgM12 binds with high affinity to complex gangliosides GD1a and GT1b. Addition of rHIgM12 to neurons in culture increases α-tubulin tyrosination levels, suggesting an alteration of microtubule dynamics. We previously reported that a single peripheral dose of rHIgM12 preserved neurologic function in a murine model of demyelination with axon loss. Because rHIgM12 improves three different models of neurologic disease, we propose the IgM may act late in the cascade of neuronal stress and/or death by a broad mechanism.